Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — BioCapital Europe 2021
21 slides from Pharming Group Nv's conference presentation, Mar 11, 2021
Slide 1: Pharming Group N.V.
Pharming Group N.V. BioCapital Europe Sijmen de Vries, CEO 11 March 2021
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation may contain forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future
Slide 3: Company Overview
Company Overview Established in 1988, based in the Netherlands with 250+ employees Listed on the Nasdaq: PHAR & Amsterdam stock exchange: PHARM Rare and ultra-rare disease development and commercialisation: • Marketed lead product: RUCONEST® (rhC1INH) • Recombinant human C1-esterase inhibitor (enzym
Slide 4: Financial highlights
Financial highlights • Record revenue in FY 2020, 9.9% increase to €185.7m (FY 2019: €169.0m) • Gross profits in FY 2020 increased 11.8% to €165.1m (FY 2019: €147.7m) • Operating profit in FY 2020 increased 10.7% to €67.4m (FY 2019: €60.9m) • Net profit in FY 2020 decrease of 9.8% to €32.7m (FY 2019
Slide 5: Investing for long-term sustainable revenue growth
Investing for long-term sustainable revenue growth
Slide 6: Three-pillar strategy for growth
Three-pillar strategy for growth Continuing to grow RUCONEST® sales Expanding indications In-licensing or through further country for rhC1INH & developing acquiring late-stage launches & increasing new recombinant proteins clinical development HAE market share using our platform candidates technolog
Slide 7: RUCONEST® in a changing (US) HAE landscape
RUCONEST® in a changing (US) HAE landscape • RUCONEST® approved for the treatment of acute HAE attacks in adults and children • Patients’ treatment plans (if on prophylaxis) include break-through medication ▪ New prophylactic treatments offer better attack reduction rates than previous IV plasma-der
Slide 8: Investment to increase capacity due to strong demand
Investment to increase capacity due to strong demand • Investment in de-risking and upscaling of production capacity ◦ Pharming received both EMA and FDA approval for its new production facility of starting material for RUCONEST® ◦ Third facility under construction to safeguard future growth in HAE
Slide 9: New opportunities for rhC1INH
New opportunities for rhC1INH • Clinical trial for rhC1INH in pre-eclampsia and acute kidney injury temporarily halted due to COVID-19 • Clinical trials for rhC1INH in patients hospitalized with confirmed SARS-CoV-2 infections ◦ University Hospital of Basel, Basel, Switzerland ▪ Results from compass
Slide 10: leniolisib – a late-stage product for APDS
leniolisib – a late-stage product for APDS APDS market leniolisib program • Activated PI3 kinase delta syndrome (APDS) is ultra-rare primary • 'navigateAPDS' partnership in the US and Canada immunodeficiency (PID) ◦ Collaboration with Invitae for a commercially ◦ Caused by autosomal dominant mutatio
Slide 11: Impact of COVID-19 on Pharming’s business
Impact of COVID-19 on Pharming’s business Pharming continues to comply with international guidance and requirements across its operations to prioritise the health and safety of its employees during the COVID-19 pandemic. The impact of COVID-19 on the operations of the business is summarized below: •
Slide 12: Outlook for 2021
Outlook for 2021 • Continued growth in revenues from sales of RUCONEST®, mainly driven by the US and expanded EU operations, subject to the progression of the COVID-19 pandemic, with quarterly fluctuations in revenues expected, as a result of the ongoing effects of the pandemic on access to customer
Slide 13: www.pharming.com ENXTAM: PHARM Nasdaq: PHAR
www.pharming.com ENXTAM: PHARM Nasdaq: PHAR Bloomberg: PHAR.AS
Slide 14: rhC1INH and severe complications of COVID-19 infections
rhC1INH and severe complications of COVID-19 infections C1INH is a protein that naturally occurs in the human body. It regulates several inflammatory pathways in the body by inhibiting certain proteins that are part of the human immune system. • In diseases like HAE, deficiency of functional C1 inhi
Slide 15: Average Clinical Trial Trajectory
Average Clinical Trial Trajectory Approval & Preclinical Phase 1 Phase 2 Phase 3 commercialisation Basic research Typically enrolling 20-100 Approximately 70% of all Approximately 33% of Only 25 to 30% of Drug discovery volunteers or mildly new drug research new drug research treatments are approved
Slide 16: RUCONEST®: Patient Segmentation in HAE
RUCONEST®: Patient Segmentation in HAE Convenience Market (Prophylactic therapies) Convenience is not as important for this group, for whom it’s all Breakthrough about efficacy and reliability Attacks RUCONEST® is the only # of HAE recombinant protein replacement patients therapy for HAE, and serves
Slide 17: Clinical trials: COVID-19 (Basel + Mexico & Brazil)
Clinical trials: COVID-19 (Basel + Mexico & Brazil) Conestat Alfa in the Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19 • Investigator initiated study: University Hospital • Phase 2 trial Basel, Basel, Switzerland • Up to 150 participants • Led by: Prof. Michael Ost
Slide 18: Clinical trials: COVID-19 (US)
Clinical trials: COVID-19 (US) Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19 • Pharming Technologies B.V. study • Phase 2 trial • 120 participants • Estimated start date: September 15, 2020 in • Randomised, open-label, parallel-group, multiple centers in the US con
Slide 19: Clinical trials: leniolisib (APDS)
Clinical trials: leniolisib (APDS) Study of Efficacy of Leniolisib in Patients With APDS/PASLI + Extension study • Novartis Pharmaceuticals in partnership with Pharming Outcome: over 12-week timeframe Technologies B.V. study • Part 1 safety, tolerability, dose PD and PK/PD • Start date: August 24, 2
Slide 20: Clinical trials: Pre-eclampsia
Clinical trials: Pre-eclampsia Study of Efficacy in the treatment of patients with Pre-Eclampsia with rhC1 INH (conestat alfa). • Pharming Technologies B.V. study • Phase 1/2 trial • 30 participants with mild/late stage pre- • Start date: on hold due to COVID-19 eclampsia • Recruiting status: Recrui
Slide 21: Clinical trials: Acute Kidney Injury
Clinical trials: Acute Kidney Injury Study to evaluate the efficacy of conestat alfa compared to placebo after PCI in NSTEMI patients. • Following positive results from a Phase 2 • Phase 2 trial investigator-initiated study of RUCONEST® in a • 220 participants double-blind, placebo-controlled clinic